The US Food and Drug Administration (FDA) has approved Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF).

The company announced the approval for the drug as a treatment for patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation.

The approval marks the first time that people with CF ages 12 years and older who have one F508del mutation and one minimal function mutation will have a medicine that targets the underlying cause of their CF. It also means that approximately 6,000 potential patients will be able to receive the treatment, and a further 12,000 who are currently eligible for one of Vertex’s three other FDA-approved CF medicines are now also eligible for Trikafta.

The approval “marks a milestone for CF patients, their families and Vertex,” explained Jeffrey Leiden, Vertex's chairman, president and chief executive officer.

He continued, “After a 20-year journey together, we have received FDA approval of Trikafta: a single breakthrough medicine with the potential to treat up to 90% of all people with CF in the future. For approximately 6,000 people with CF in the US, Trikafta is the first medicine that can treat the underlying cause of their disease.”

“I want to personally thank the hundreds of Vertex scientists who have been working on this program for nearly 20 years – many of whom have dedicated their entire careers to changing the course of this disease; the CF Foundation which has provided support, encouragement and help throughout the journey; and most importantly the thousands of patients, caregivers, doctors and advocates who have courageously and persistently worked side-by-side with us to get to where we are today.”

Vertex has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the treatment, and is currently evaluating it in people ages six through 11 with F/MF and F/F CF mutations in an ongoing Phase III study.